Researchers Show Feasibility of Stem Cell-Derived Organoids for Developing Gene Therapy Vectors

June 15, 2018

(News Medical) – A new study that compared six of the most promising adeno-associated viral (AAV) gene therapy vectors in human retinal organoid models showed clear distinctions in the efficiency of gene transfer to both retinal pigment epithelial (RPE) and photoreceptor cells. The results of this study can help guide future selection and design of viral vectors for therapeutic gene delivery and gene editing and are reported in an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.